uniQure: FDA tidak lagi setuju data Fase I/II cukup untuk BLA AMT-130

FDA membalikkan panduan sebelumnya dan kini menyatakan bahwa data Fase I/II dibandingkan kontrol eksternal mungkin tidak memadai sebagai bukti utama untuk pengajuan BLA. Waktu pengajuan BLA yang direncanakan untuk AMT-130 kini tidak jelas. uniQure akan meminta risalah rapat akhir dan diskusi tindak lanjut mendesak dengan FDA untuk mengidentifikasi langkah ke depan.

Fakta utama

Diajukan: 3 Nov 2025, 12.06
Peristiwa: Lainnya
Arah: Negatif
Perusahaan: QUREuniQure
Aset: AMT-130
Sumber: SEC 8-K
Keyakinan: Bersumber

Kutipan sumber

EX-99.1 2 qure-20251103xex99d1.htm EX-99.1 Exhibit 99.1 uniQure Provides Regulatory Update on AMT-130 for Huntington ’ s Disease Lexington, MA and Amsterdam, the Netherlands, November 3, 2025 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it received feedback from the U.S. Food and Drug Administration (FDA) during a recent pre-Biologics License Application (BLA) meeting regarding AMT-130, an investigational gene therapy for Huntington ’ s disease (HD). Though final meeting minutes have not yet been received, based on the discussions at the meeting, uniQure believes that the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 in comparison to an ex

SEC 8-K